Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials. For resources pertaining to regenerative cell therapy and clinical treatment, check out R3 Stem Cell.

Posted on: 4 January 2025, source: pmlive.com
Novartis has shared positive top-line results from a late-stage study of its investigational gene therapy in patients aged two to less than 18 years with the rare neuromuscular disease spinal muscular atrophy (SMA). The phase 3 STEER study has been evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with SMA type 2 who were able to sit but had never walked independently. Results were compared against a sham control, which is designed to mimic the administration of an investigational drug without delivering any active treatment.

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Posted on: 17 December 2024, source: Indian Express
In a significant medical breakthrough, India’s first human gene therapy with a lentiviral vector for severe haemophilia-A has been found to be successful with zero bleeding rate in patients. Haemophilia is a serious bleeding disorder caused by blood deficiency of clotting factors (factor VIII and factor IX proteins). It can lead to spontaneous bleeding episodes, joint damage, and a reduced quality of life for patients and their families. The new gene therapy has shown promising results during the first phase trial on five patients, aged between 22 and 41. The annualised bleeding rate has been observed to be zero in all five patients over a cumulative follow-up of 81 months. Supported by the Department of Biotechnology, this first-in-human gene therapy for haemophilia-A patients, has been developed by Centre for Stem Cell Research (CSCR) at CMC, Vellore.

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Posted on: 25 November 2024, source: medcitynews.com
A patient death in a clinical trial usually prompts the FDA to impose a clinical hold. But Neurogene is able to avoid a lengthy clinical trial pause largely because of its participation in an FDA pilot program intended to speed up the development of therapies for rare diseases. The clinical trial participant who was hospitalized for severe complications after receiving the high dose of Neurogene’s experimental Rett syndrome gene therapy has died. Neurogene disclosed the death in a regulatory filing after the market close Thursday. The company said the FDA is permitting the Phase 1/2 study to continue with the low dose of the therapy, code-named NGN-401. Neurogene added that it will incorporate this dose in planning the design of the clinical trial expected to support a regulatory submission seeking FDA approval of the gene therapy.

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Posted on: 30 October 2024, source: Labroots
Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where they perform their gene editing function. In this approach, a nanoparticle exposes cells to the instructions for building CRISPR reagents and the correct gene sequence to repair a gene defect. The method also aims to repair genetic problems at very early stages of development. While more research will be needed, the investigators are hopeful that this technique will eventually be used to treat human patients who are identified during prenatal testing.

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Posted on: 12 October 2024, source: pharmaceutical-technology.com
The side effects of bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) have been thrust back into the spotlight after new data shows seven children who took part in its clinical studies went on to develop a type of blood cancer. The findings, from a study published in The New England Journal of Medicine (NEJM) yesterday (9 October), show that seven out of 67 patients under 18 years of age who took part in a Phase II and Phase III trial for Skysona developed haematologic cancers. The patients received the one-time autologous hematopoietic stem cell-based gene therapy as a treatment for early cerebral adrenoleukodystrophy (CALD) – a rare and fatal neurodegenerative disease.

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Posted on: 1 October 2024, source: prnewswire
The ISCT Institute of Training & Development, a division of the International Society for Cell and Gene Therapy (ISCT), has become the first cell and gene therapy (CGT) organization to receive globally recognized accreditation from the ANSI National Accreditation Board (ANAB) for excellence in workforce development and training. This prestigious accreditation underscores ISCT's leadership in addressing critical skill gaps within the rapidly evolving CGT sector, while ensuring that ISCT's courses are standardized, globally recognized, and accredited.

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Posted on: 26 August 2024, source: fabrydiseasenews.com
The first patient has been dosed in uniQure’s U.S. Phase 1/2a clinical trial of AMT-191, its gene therapy candidate for Fabry disease. The clinical trial, an open-label study (NCT06270316) focused on safety and early efficacy, is still recruiting about 12 adult male patients at a site in Fairfax, Virginia. Eligible men will have had a suboptimal response to enzyme replacement therapy (ERT). An open-label trial means that both researchers and participants know what treatment is being administered.

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